IB1001

BIOLOGICAL6 trials

Sponsors

Medexus Pharma, Inc., Cangene Corporation, IntraBio Inc

Conditions

Ataxia TelangiectasiaGM2 GangliosidosisHemophilia BNiemann-Pick Disease, Type CSandhoff DiseaseTay-Sachs Disease

Phase 2

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

CompletedNCT00768287

Medexus Pharma, Inc.Hemophilia B

Start: 2009-01-31End: 2016-12-31Updated: 2021-04-06

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

CompletedNCT03759665

IntraBio IncGM2 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease

Start: 2019-06-07End: 2023-01-09Updated: 2024-03-12

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

TerminatedNCT03759678

IntraBio IncAtaxia Telangiectasia

Start: 2020-01-08End: 2025-10-27Updated: 2026-03-25

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

CompletedNCT03759639

IntraBio IncNiemann-Pick Disease, Type C

Start: 2019-09-04End: 2022-11-07Updated: 2023-11-28

Phase 3

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

TerminatedNCT01271868

Medexus Pharma, Inc.Hemophilia B

Start: 2010-11-30End: 2016-12-31Updated: 2021-03-15

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

WithdrawnNCT02048111

Cangene CorporationHemophilia B

Start: 2014-03-31End: 2015-11-30Updated: 2016-08-23

Related Papers

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses

Journal of Neurology2026-07-17

Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses.

2022-12-0123 citations

Effects of Acetyl-DL-Leucine on Ataxia and Downbeat-Nystagmus in Six Patients With Ataxia Telangiectasia

Journal of Child Neurology2021-10-0715 citations

Efficacy and safety of N-acetyl-l-leucine in Niemann–Pick disease type C

Journal of Neurology2021-08-1357 citations

A master protocol to investigate a novel therapy acetyl-l-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia

Trials2021-01-2234 citations

Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis.

2012-07-0521 citations